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EP 4 269 587 A3 |
(12) |
EUROPEAN PATENT APPLICATION |
(88) |
Date of publication A3: |
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10.07.2024 Bulletin 2024/28 |
(43) |
Date of publication A2: |
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01.11.2023 Bulletin 2023/44 |
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Date of filing: 19.09.2017 |
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International Patent Classification (IPC):
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Designated Contracting States: |
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AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL
NO PL PT RO RS SE SI SK SM TR |
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Priority: |
19.09.2016 ES 201631216
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(62) |
Application number of the earlier application in accordance with Art. 76 EPC: |
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17776976.7 / 3516059 |
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Applicants: |
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- Universitat de València
46010 Valencia (ES)
- AUM LifeTech, Inc.
Philadelphia, PA 19104 (US)
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(72) |
Inventors: |
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- ARTERO ALLEPUZ, Rubén D.
46100 Burjassot, Valencia (ES)
- LLAMUSÍ TROISI, María Beatriz
46100 Burjassot, Valencia (ES)
- CERRO HERREROS, Estefanía
46100 Burjassot, Valencia (ES)
- FERNÁNDEZ COSTA, Juan M.
46100 Burjassot, Valencia (ES)
- AISHWARYA, Veenu
Philadelphia, 19104 (US)
- MØLLER, Thorleif
5792 Arslev (DK)
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Representative: Hoffmann Eitle |
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Hoffmann Eitle S.L.U.
Paseo de la Castellana 140, 3a planta
Edificio LIMA 28046 Madrid 28046 Madrid (ES) |
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(54) |
MODULATION OF MICRORNAS AGAINST MYOTONIC DYSTROPHY TYPE 1 AND ANTAGONISTS OF MICRORNAS
THEREFOR |
(57) Modulation of microRNAs against myotonic dystrophy type 1 and antagonists of microRNAs
therefor. The invention provides the use of inhibitors of microRNAs repressors of
MBNL1 and/or MBNL2 genes for the manufacture of a medicinal product for the treatment
of myotonic dystrophy 1. Inhibiting these microRNAs allows to increase the endogenous
levels of the corresponding proteins MBNL1 and/or MBNL2, thereby alleviating symptoms
of the disease, especially when inhibiting repressors that are expressed in the main
affected organs: skeletal muscle, heart or organs of the central nervous system. The
inhibition of the microRNAs miR-23b-3p and miR-218-5p is preferred. It also provides
oligoribonucleotide or oligoribonucleotide analogue antagonists suitable therefor,
preferably antagomiRs directed against the microRNAs mentioned with chemical modifications
that enhance their interaction with the target, their stability in vivo and their
ability to penetrate into the cells and distribute throughout tissues and organs.